An Integrated Approach to Non-Viral Gene Therapy for Serious and Rare Diseases

SmartPharm Therapeutics is a second-generation gene-encoded therapeutics company that has benefited from the first decades of learning in gene therapy development to produce an evolved therapeutic platform. We integrate these insights with key technologies and engineering capabilities to enable us to produce optimal gene-encoded therapeutics for patients.

Biologics from within

“At SmartPharm, our vision is to transform the nature of biologics therapy to improve effectiveness of therapy, enhance patient quality of life, and extend the global reach of such treatments.”

SmartPharm is focused on “gene-encoded therapeutics”

Three Ways to Deliver Therapeutic Proteins: pDNA can be inserted into bacteria or Chinese hamster Ovary (CHO) cells to make recombinant proteins given to people; mRNA or pDNA can be delivered ex vivo to human cells capable of making the proteins in the patient (cell therapy). mRNA or pDNA is delivered to human tissues that make proteins in the body (gene encoded therapy).

The bulk of modern biologics therapy is built on gene engineering. In traditional biologics therapy, genes are inserted into bacterial, fungal, or human cell lines to make different kinds of therapeutic proteins for use in patients. More recently, human cells outside the body have been genetically altered and then delivered to patients as therapy (cell therapy). Gene therapy is where the genetic alteration of human cells is performed inside the body. The most recent offshoot of this approach is to deliver a gene-editing platform, such as TALEN or CRISPR/Cas9 into cells to alter the genome of a patient for therapeutic purposes (genomic therapy). Other gene therapy approaches use nucleic acids to turn off unwanted or abnormal proteins inside cells, shut down specific cellular processes, and even to make unwanted cells (like cancer cells) undergo programmed cell death.

A subset of gene therapy seeks to insert nucleic acids (mRNA or DNA) into cells to make normal versions of a missing or abnormal program or to produce an entirely new type of protein that has therapeutic benefit. This approach eliminates the need for administering a recombinant therapeutic protein—the body itself becomes the factory for protein production. SmartPharm Therapeutics is focused on this approach to gene therapy, which we call “gene-encoded therapeutics.”